Real-World Evidence in FDA Approvals for Labeling Expansion of Small Molecules and Biologics.

ABSTRACT:

Introduction

Real-world evidence (RWE) can support the evaluation of safety and efficacy for medical products, but its extent of use in labeling expansion submissions remains unclear. This study aimed to characterize the RWE used in labeling expansion or likely used, as identified through literature search, for drugs and biologics.

Methods

We identified RWE used in FDA-approved labeling expansion for drug and biologic supplemental applications from January 2022 to May 2024 (Drugs@FDA), using FDA prescribing information and review documents. We also searched ClinicalTrials.gov and PubMed to identify RWE not included in the FDA approval letter and labeling but could have been incorporated into submissions by sponsors. Characteristics of the RWE were extracted and summarized.

SUBMITTER: Deng YF 

PROVIDER: S-EPMC12446098 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature